BOSTON — Eculizumab significantly reduced disease severity and improved quality of life at 26 weeks in adolescents with refractory generalized myasthenia gravis, according to a presenter at the American Academy of Neurology annual meeting.“[It is important] to boost the knowledge of the community to have interventional data in a targeted myasthenia therapy,” John F. Brandsema,
Alexion, AstraZeneca Rare Disease, today announced that Health Canada has granted a Notice of Compliance (NOC) for KOSELUGO (selumetinib) for the treatment of pediatric patients aged two years and above, with neurofibromatosis type 1 (NF1) who have.
Addition of ravulizumab to BSC did not improve survival or other secondary outcomes.
Safety findings were consistent with the known safety profile of ravulizumab in its
approved indications. Despite the lack of efficacy, the study adds value for future
research into complement therapeutics in critical illnesses by showing that C5 inhibition
can be accomplished in severely ill patients.
PARIS, FRANCE, Feb. 28, 2023 (GLOBE NEWSWIRE) On Rare Disease Day, Ad Scientiam, a leader in digital biomarkers, announced the launch of two ambitious programs to develop and validate novel digital